⚡Three FDA dermatology approvals

Good morning — it’s Saturday, October 4, and we’re covering the first FDA-approved BTK inhibitor for chronic spontaneous urticaria, two new pediatric dermatology approvals, and more.

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Novartis’ remibrutinib (Rhapsido) has become the first Bruton’s tyrosine kinase (BTK) inhibitor approved by the FDA for adults with chronic spontaneous urticaria uncontrolled by H1 antihistamines.

The approval introduces the only oral alternative to the injectable biologics omalizumab (Xolair) and dupilumab (Dupixent).

BTK acts downstream of the high-affinity IgE receptor (FcεRI) on mast cells and basophils, leading to the release of histamine and other proinflammatory mediators. It also drives B-cell autoantibody production. By selectively inhibiting BTK, remibrutinib interrupts this pathway.

The FDA’s decision was supported by the twin REMIX-1 and REMIX-2 Phase 3 trials (n=925), recently published in the NEJM.

By Week 24, 52–55% of patients on remibrutinib 25 mg twice daily achieved well-controlled disease (UAS7 ≤6) vs 28–35% on placebo (P<0.001), with 36% of remibrutinib-treated patients experiencing complete symptom resolution (UAS7=0). Separation from placebo emerged as early as Week 1.

Long-term extension data showed further benefit, with nearly half reaching complete clearance at one year.

Remibrutinib showed a favorable safety profile without the need for laboratory monitoring. During the double-blind period, adverse events were comparable to placebo. Petechiae occurred more often with treatment (3.8% vs 0.3%) but led to discontinuation in only one patient. Exposure-adjusted event rates remained stable in the long-term extension.

Novartis has filed regulatory submissions globally, including in the EU, Japan, and China. U.S. pricing is set at $4,521 per month. Meanwhile, Sanofi is also advancing another BTK inhibitor, rilzabrutinib, in chronic spontaneous urticaria, suggesting this mechanism may become a new treatment class for the condition.

The FDA has expanded approval of Incyte’s ruxolitinib cream (Opzelura) to children aged 2–11 with mild-to-moderate atopic dermatitis. Ruxolitinib cream becomes the first topical JAK inhibitor available for this age group.

The Phase 3 TRuE-AD3 trial supported the approval. In stage 1 of the trial, investigators randomized 330 children with 3%–20% body surface area involvement to twice-daily ruxolitinib cream (0.75% or 1.5%) or vehicle for 8 weeks.

Clear or almost clear skin (IGA 0/1 with ≥2-point improvement) was achieved by 57% of patients on the higher ruxolitinib dose and 37% on the lower dose compared with 11% on vehicle (P<0.0001, P=0.0001). Improvements were evident as early as Week 2.

In stage 2 of the trial, all children received an additional 44 weeks of as-needed ruxolitinib treatment: those already on ruxolitinib continued their dose, while those on vehicle were re-randomized to 0.75% or 1.5%.

Responses were sustained through Week 52, with patients remaining off treatment nearly half the time because their lesions had cleared.

Both doses were well tolerated. Application-site reactions were uncommon (≤5%), even though 64% of patients had facial or neck involvement. No adverse events suggested systemic JAK inhibition, consistent with low plasma concentrations across age groups.

Ruxolitinib now joins tapinarof (Vtama) and roflumilast (Zoryve) as newer nonsteroidal topical options for pediatric atopic dermatitis. It is also approved for nonsegmental vitiligo in patients aged 12 years and older.

> Tremfya secures pediatric approvals for psoriasis and psoriatic arthritis: The FDA has also approved guselkumab (Tremfya) for children aged 6 years and older with moderate-to-severe plaque psoriasis or active psoriatic arthritis. The biologic becomes the only IL-23 inhibitor approved for these pediatric indications. The decision was supported by the Phase 3 PROTOSTAR trial, a study of 120 patients aged 6–18 years with disease inadequately controlled by phototherapy or topical therapy. At Week 16, significantly more patients on guselkumab achieved clear or almost clear skin (IGA 0/1) than those on placebo (66% vs 16%; P<0.001), and responses further improved through one year of therapy. Safety findings were consistent with adult Phase 3 data. The FDA granted the psoriatic arthritis indication based on pharmacokinetic extrapolation from adjacent adult and pediatric trials. Approved pediatric dosing is 100 mg SC at Weeks 0 and 4, then every 8 weeks for children aged 6 years and older who weigh at least 40 kg. Guselkumab, a dual-acting antibody, blocks IL-23 and engages CD64 on myeloid cells that produce IL-23. With one-third of psoriasis cases beginning in childhood, these approvals mark a milestone in pediatric psoriasis care.

> Affibody advances izokibep with favorable Phase 3 data in hidradenitis suppurativa: Affibody presented positive 16-week Phase 3 results for izokibep in hidradenitis suppurativa as a late-breaker at EADV 2025. At one-tenth the size of a monoclonal antibody, izokibep potently inhibits IL-17A. Its compact size, which enhances tissue penetration, and albumin-binding domain together improve pharmacokinetics. Investigators randomized 258 adults with moderate-to-severe disease to weekly subcutaneous izokibep 160 mg or placebo. At Week 16, 37% of izokibep-treated patients achieved HiSCR75 (≥75% reduction in abscess and nodule counts with no increase in abscesses or draining tunnels) compared with 20% on placebo (P<0.01). Higher-order endpoints also favored izokibep, including HiSCR90 (24% vs 12%; P<0.05) and HiSCR100 (21% vs 9%; P<0.01). HiSCR50 rates were 50% vs 32% (P<0.01). Safety was reassuring. Most adverse events were mild, and serious events were infrequent (1% vs 4% on placebo). Notably, izokibep resulted in no cases of inflammatory bowel disease or candidiasis, adverse events reported with dual IL-17A/F inhibitors. Although cross-trial comparisons have limitations, izokibep’s efficacy appears at least comparable to the IL-17 blockers bimekizumab and secukinumab.

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